US2021130429A1PendingUtilityA1

Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases

Assignee: UNIV SOUTH FLORIDAPriority: Jul 1, 2011Filed: Oct 27, 2020Published: May 6, 2021
Est. expiryJul 1, 2031(~4.9 yrs left)· nominal 20-yr term from priority
C07K 14/7158C12N 2750/14171A61K 48/00A61K 38/00C12N 2750/14143C07K 14/521
49
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Claims

Abstract

The subject invention pertains to the use of fractalkine (FKN, CX3CL1) and its receptor CX3CR1 for treatment of neuroinflammation and/or neurodegeneration. In one embodiment, the present invention provides a method for treatment of neuroinflammation and/or neurodegenerative diseases, comprising: administering, to cells of a subject in need of such treatment, an adeno-associated virus that comprises a functional fractalkine gene operably linked to transcriptional control elements. In one embodiment, the subject invention is used to treat or ameliorate Parkinson's disease. The present invention can also be used to treat or ameliorate neuroinflammatory and/or neurodegenerative diseases including, but not limited to, Alzheimer's disease, epilepsy, aging, and traumatic brain injury.

Claims

exact text as granted — not AI-modified
1 - 12 . (canceled) 
     
     
         13 . A recombinant adeno-associated virus (rAAV) vector comprising a nucleic acid molecule encoding a fractalkine peptide operably linked to a promoter. 
     
     
         14 . The rAAV vector of  claim 13 , wherein the fractalkine peptide is a soluble isoform of the fractalkine peptide or is a fractalkine peptide that can be endogenously cleaved into a soluble isoform of the fractalkine peptide. 
     
     
         15 . The rAAV vector of  claim 13 , wherein the fractalkine peptide has an amino acid sequence having at least 85% identity to any of SEQ ID NOs: 6, 7, 9, or 10. 
     
     
         16 . A pharmaceutical composition comprising the rAAV vector of  claim 13 , formulated with pharmaceutically acceptable carriers. 
     
     
         17 - 20 . (canceled) 
     
     
         21 . The rAAV vector of  claim 13 , wherein the rAAV9 vector. 
     
     
         22 . The rAAV vector of  claim 13 , wherein the promoter is a nervous tissue cell-specific promoter.

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